Lipid-like materials for low-dose, in vivo gene silencing
Open accessAbstract
Significant effort has been applied to discover and develop vehicles which can guide small interfering RNAs (siRNA) through the many barriers guarding the interior of target cells. While studies have demonstrated the potential of gene silencing in vivo, improvements in delivery efficacy are required to fulfill the broadest potential of RNA interference therapeutics. Through the combinatorial synthesis and screening of a different class of materials, a formulation has been identified that enables siRNA-directed liver gene silencing in mice at doses below 0.01 mg/kg. This formulation was also shown to specifically inhibit expression of five hepatic genes simultaneously, after a single injection. The potential of this formulation was further validated in nonhuman primates, where high levels of knockdown of the clinically relevant gene transthyretin was observed at doses as low as 0.03 mg/kg. To our knowledge, this formulation facilitates gene silencing at orders-of-magnitude lower doses than required by any previously described siRNA liver delivery system.
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Considerable effort to reduce siRNA payload by developing different lipid formulations has led to a tenfold or even 100-fold reduction in the dose of siRNA administered to different models.
“considerable effort has been made to reduce the siRNA payload by developing different lipid formulations for delivery, which has so far led to a tenfold or even 100-fold reduction in the dose of siRNA administered to different models77,78.”
From Perspectives and opportunities for nanomedicine in the management of atherosclerosis (2011) · cites this paper as [78]
All these lipid materials showed improved delivery efficiency compared with DLin-KC2-DMA LNPs.
“All these lipid materials showed improved delivery efficiency as compared with DLin-KC2-DMA LNPs.[38, 64, 65]”
From RNA delivery biomaterials for the treatment of genetic and rare diseases (2019) · cites this paper as [65]
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Self-citationVisualization of self-delivering hydrophobically modified siRNA cellular internalization
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Biodegradable Amino-Ester Nanomaterials for Cas9 mRNA Delivery in Vitro and in Vivo
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- Authors
- Love, K. T., Mahon, K. P., Levins, C. G., Whitehead, K. A., Querbes, W., Dorkin, J. R., Qin, J., Cantley, W., Qin, L. L., Racie, T., Frank-Kamenetsky, M., Yip, K. N., Alvarez, R., Sah, D. W. Y., de Fougerolles, A., Fitzgerald, K., Koteliansky, V., Akinc, A., Langer, R., & Anderson, D. G.
- Affiliations
- Massachusetts Institute of Technology (US)
- Koch Institute for Integrative Cancer Research At MIT (US)
- Harvard University (US)
- Alnylam Pharmaceuticals (United States) (US)
- Alnylam Pharmaceuticals, Inc., 300 Third Street, Cambridge, MA 02142,
- Department of Chemical Engineering, Massachusetts Institute of Technology, Cambridge, MA 02139,
- ORCID
- Venue
- Proceedings of the National Academy of Sciences
- Publisher
- National Academy of Sciences
- Published
- 2010-01-11
- Volume
- 107
- Issue
- 5
- Pages
- 1864-1869
- ISSN
- 0027-8424 (ISSN-L)
- 1091-6490
- PMC
- PMC2804742
- OpenAlex
- W2089150369
- Cited by
- 969
- References
- 34
- Citations by year
- 2026: 45 · 2025: 67 · 2024: 75 · 2023: 65 · 2022: 59 · 2021: 68 · 2020: 62 · 2019: 48 · 2018: 42 · 2017: 45 · 2016: 66 · 2015: 53 · 2014: 67 · 2013: 54 · 2012: 77
- FWCI
- 26.86
- Type
- journal article
- Language
- en
- Update notices
- correction (2010-05-05)
- Open access
- green
- www.ncbi.nlm.nih.gov
- Locations
- Proceedings of the National Academy of Sciences (publishedVersion)
- PubMed Central (publishedVersion)
- PubMed (publishedVersion)
- DSpace@MIT (Massachusetts Institute of Technology) (submittedVersion, cc-by-nc)
- Funders
- Alnylam Pharmaceuticals
- National Institutes of Health (4r37eb000244-34)
- National Institute of Biomedical Imaging and Bioengineering (EB-000244, R01EB000244, R37-EB000244)
- Topics
- Life Sciences › Biochemistry, Genetics and Molecular Biology › Molecular Biology › RNA Interference and Gene Delivery
- Physical Sciences › Materials Science › Biomaterials › Nanoparticle-Based Drug Delivery
- Life Sciences › Biochemistry, Genetics and Molecular Biology › Molecular Biology › RNA and protein synthesis mechanisms
- Keywords
- siRNA delivery, RNA interference therapy, prealbumin
- MeSH terms
- Animals, Biocompatible Materials, Factor VII, HeLa Cells, Humans, Lipids, Macaca fascicularis, Mice, Inbred C57BL, Molecular Structure, Drug Delivery Systems, Gene Silencing, Hepatocytes, RNA Interference, RNA, Small Interfering, Mice
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Registry, retraction, Cites and statistics results are computed, not judged, with one exception: a printed reference that no index lists is read once by a model before it counts as a citation error. Claims are extracted by a model and kept only when their quote appears verbatim in the text; each cited source is then checked, with a model’s help, for whether it says what the claim relies on. Those verdicts can be wrong — read the claim’s quote and the cited source before relying on one.
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